Human Stem Cell Use: iPS Cell


hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders

Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders. Impact This proposal addresses a significant unmet clinical need for a cell therapy approach for gastrointestinal motility disorders such as Hirschsprung disease, achalasia and gastroparesis. […]

Defining the source of dysfunction in monogenic Intellectual Disability Syndrome neurons

Research Objective This study will use pluripotent stem cells derived from patients to determine why Intellectual Disabilities caused by mutations in chromatin regulatory proteins leads to neuronal defects. Impact Our study of intellectual disability syndromes will determine links between mutations and neuronal dysfunction Major Proposed Activities Our study of intellectual disability syndromes will determine links […]

Characterization and applications of human blastoids for understanding early human embryogenesis

Research Objective Our work will yield an improved stem-cell based embryo model that we will explore with various omics approaches and genetic screens to gain insights into the pathways that control human embryos. Impact An improved stem cell-based embryo model is a crucial step for in-depth studies of human development and will enhance our ability […]

Modeling Retinitis Pigmentosa using patient-derived human iPSC organoids

Research Objective The objective of this proposal is to develop a human retinal organoid model of adRP to gain insights in pathogenesis and assess clinically relevant approaches to restore RHO protein function. Impact Upon successful completion of this study, we will have established a disease-in-a-dish model and a novel therapeutic approach towards management of the […]

Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis

Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS disease phenotypes Impact Our goal is to determine if ataxin-3 genetic dysregulation is a target for the development of therapies to treat ALS (Lou Gehrig's […]

Identifying roadblocks to neural stem cell transplantation into human tissues.

Research Objective We will generate a comprehensive map of human neural stem cell differentiation profiles that will serve as a reference for enhancing neural stem cell-based therapies. Impact Our project will develop improved protocols for human neural stem cells differentiation, enhancing the fidelity, safety and robustness of future cell therapies. Major Proposed Activities Establish quantitative […]

Functional genomics to study cellular convergence across ASD risk genes in neurodevelopment

Research Objective Our objective is to enable scalable genetic screening to study how neurogenesis is impacted by risk genes implicated in human psychiatric disorders. Impact We will develop and apply state-of-the-art genomic analysis to seek mechanisms and disease modifying solutions. Major Proposed Activities Identifying and validating efficient gRNA for gene editing Identify risk gene effects […]

Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies

Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]

Establishment of a novel approach to systematically study the dynamic organization of protein complexes in stem cells

Research Objective We focus on hiPSCs pluripotency and neurodifferentiation to develop a novel framework to allow simultaneous identification of multiple interactions between proteins and between proteins and the genome Impact Our framework will allow high-throughput queries of the organization and functionality of proteins and shift the focus towards unprecedented, multi-dimensional studies of the cellular complexity. […]

Understanding Chemotherapy-Induced Peripheral Neuropathy Mechanisms using CRISPRi and Chemical Screens in Human iPSC-Derived Sensory Neurons

Research Objective The research objectives are to identify causal genes for chemotherapy-induced mitochondrial toxicity and neurodegeneration in sensory neurons and drugs that target this toxicity. Impact These studies will open the possibility for genetic or drug targeting to prevent and treat drug-induced peripheral neuropathies and possibly neuropathies caused by disease or inherited. Major Proposed Activities […]

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